S. HRG. 115–663 GENE EDITING TECHNOLOGY: INNOVATION AND IMPACT HEARING OF THE COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS UNITED STATES SENATE ONE HUNDRED FIFTEENTH CONGRESS FIRST SESSION ON EXAMINING GENE EDITING TECHNOLOGY, FOCUSING ON INNOVATION AND IMPACT NOVEMBER 14, 2017 Printed for the use of the Committee on Health, Education, Labor, and Pensions ( Available via the World Wide Web: http://www.govinfo.gov U.S. GOVERNMENT PUBLISHING OFFICE 27–682 PDF WASHINGTON : 2019 COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS LAMAR ALEXANDER, Tennessee, Chairman MICHAEL B. ENZI, Wyoming PATTY MURRAY, Washington RICHARD BURR, North Carolina BERNARD SANDERS (I), Vermont JOHNNY ISAKSON, Georgia ROBERT P. CASEY, JR., Pennsylvania RAND PAUL, Kentucky AL FRANKEN, Minnesota SUSAN M. COLLINS, Maine MICHAEL F. BENNET, Colorado BILL CASSIDY, M.D., Louisiana SHELDON WHITEHOUSE, Rhode Island TODD YOUNG, Indiana TAMMY BALDWIN, Wisconsin ORRIN G. HATCH, Utah CHRISTOPHER S. MURPHY, Connecticut PAT ROBERTS, Kansas ELIZABETH WARREN, Massachusetts LISA MURKOWSKI, Alaska TIM KAINE, Virginia TIM SCOTT, South Carolina MAGGIE WOOD HASSAN, New Hampshire DAVID P. CLEARY, Republican Staff Director LINDSEY WARD SEIDMAN, Republican Deputy Staff Director EVAN SCHATZ, Democratic Staff Director JOHN RIGHTER, Democratic Deputy Staff Director (II) CONTENTS STATEMENTS TUESDAY, NOVEMBER 14, 2017 Page COMMITTEE MEMBERS Alexander, Hon. Lamar, Chairman, Committee on Health, Education, Labor, and Pensions, opening statement ....................................................................... 1 Murray, Hon. Patty, a U.S. Senator from the State of Washington, opening statement .............................................................................................................. 3 Warren, Hon. Elizabeth, a U.S. Senator from the State of Massachusetts ........ 5 Collins, Hon. Susan M., a U.S. Senator from the State of Maine ....................... 26 Scott, Hon. Tim, a U.S. Senator from the State of South Carolina ..................... 29 Hassan, Hon. Maggie Wood, a U.S. Senator from the State of New Hamp- shire ....................................................................................................................... 31 Kaine, Hon. Tim, a U.S. Senator from the State of Virginia ............................... 37 WITNESSES Statement of Matthew Porteus, M.D., Ph.D., Associate Professor of Pediatrics, Stanford University, Palo Alto, CA .................................................................... 5 Prepared statement .......................................................................................... 7 Statement of Katrine Bosley, Chief Executive Officer and President, Editas Medicine, Cambridge, MA ................................................................................... 14 Prepared statement .......................................................................................... 16 Summary Statement ........................................................................................ 20 Statement of Jeffrey Kahn, M.D., Ph.D., Director, Johns Hopkins Berman Institute of Bioethics, Johns Hopkins School of Public Health, Baltimore, MD ......................................................................................................................... 21 Prepared statement .......................................................................................... 23 Summary Statement ........................................................................................ 25 ADDITIONAL MATERIAL Response by Matthew Porteus to questions of: Senator Collins .......................................................................................... 43 Senator Murray ......................................................................................... 44 Senator Bennet .......................................................................................... 45 Senator Whitehouse .................................................................................. 48 Response by Katrine Bosley to questions of: Senator Murray ......................................................................................... 50 Senator Casey ............................................................................................ 51 Senator Bennet .......................................................................................... 52 Senator Whitehouse .................................................................................. 53 (III) GENE EDITING TECHNOLOGY: INNOVATION AND IMPACT Tuesday, November 14, 2017 U.S. SENATE, COMMITTEE ON HEALTH, EDUCATION, LABOR, AND PENSIONS, Washington, DC. The Committee met, pursuant to notice, at 10 a.m. in room SD– 430, Dirksen Senate Office Building, Hon. Lamar Alexander, Chairman of the Committee, presiding. Present: Senators Alexander [presiding], Murray, Collins, Young, Scott, Casey, Warren, Kaine, and Hassan. OPENING STATEMENT OF SENATOR ALEXANDER The CHAIRMAN. Good morning. The Senate Committee on Health, Education, Labor, and Pen- sions will please come to order. Senator Corker is chairing a Foreign Relations Committee hear- ing down the hall about when the President of the United States can use nuclear weapons. We are taking a different tack today. We are looking at something quite different and extremely inter- esting to me. It is about gene editing and a new technology with amazing potential that raises important ethical questions as well. Senator Murray and I will each have an opening statement, then we will introduce the witnesses. After the witnesses’ testimony, Senators will each have 5 minutes of questions. Eric Lander, a leading geneticist and mathematician, who was integral to the Human Genome Project said, ‘‘It is hard to recall a revolution that has swept biology more swiftly than CRISPR.’’ Today, we are looking at this remarkable technology to edit genes that has the potential to treat devastating diseases, includ- ing those that currently have limited treatments or cures. While CRISPR is not the only way to edit the human genome, it is one of the most exciting and talked about ways in the medical research community. It is a relatively new technology. It essen- tially uses molecules that can be targeted to act as scissors to cut and edit genes. While CRISPR acts as the search function, it goes and finds the mutated gene—Cas9 is the tool that deletes the disease-causing gene—inserts new genes or repairs mutated genes. In a way, it is like cutting and pasting in a computer document. (1) 2 That may be an oversimplification, but CRISPR technology is less expensive, more precise, and more readily available to sci- entists all over the world than other gene editing technologies. A ‘‘New York Times’’ story in August reported that CRISPR can be used to do something as frivolous as making yeast glow like jel- lyfish to something as serious as making real strides against dis- eases, such as correcting the gene that causes sickle cell anemia. While CRISPR was developed in 1993, its use was perfected for humans in 2013, only 4 years ago. Its most widespread use until now has been in agriculture. Disease resistant wheat and rice has been created using CRISPR, and CRISPR has been used to modify tomatoes and soybeans to improve yields and create healthier soy- bean oil. There is the potential to create crops that can produce higher yields, are able to live through a drought, and have increased nu- tritional value. Some researchers are even looking at ways to make better tasting crops. CRISPR’s use in humans is more recent, but the possibility of the diseases it could treat, and the lives that could be improved, is remarkable. According to the Centers for Disease Control and Prevention, sickle cell disease occurs in about 1 out of every 365 African-Amer- ican births. One of our witnesses today will be able to speak to re- search on how CRISPR can help with this devastating disease. Editas Medicine, who is represented by one of our other wit- nesses today, sees the potential to treat blood disease that today are currently only treatable through blood transfusions and bone marrow transplants. Using CRISPR, the genes causing blood dis- ease could be edited and re-administered to treat the disease more safely and effectively. For cancer patients, CRISPR could improve the amount of time immune cells are active in fighting tumors. The possibilities could go on further. If we could eventually identify the gene mutation that, for exam- ple, shows a predisposition to Alzheimer’s, could we edit that gene and prevent the suffering and heartache that Alzheimer’s causes? While CRISPR, and other gene editing technologies, could trans- form human health, it is not hard to see how we can quickly get into societal and ethical issues. The technology could lead to permanent changes in the human genome. There is even the possibility of making changes in em- bryos to create so-called ‘‘designer babies.’’ In the hands of our adversaries, CRISPR poses national security concerns through the potential to produce new biological weapons. In February 2016, former Director of National Intelligence, James Clapper, added gene editing to a list of ‘‘weapons of mass destruc- tion and proliferation.’’ I know the leaders at Oak Ridge National Laboratory, and other places in the intelligence community, are having classified discus- sions similar to the one we are having today. Part of our job on this Committee is to learn about new tech- nologies, to lead the discussions with experts about the implica-